NCT01275066

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 177 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome). There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for MPS IVA patients. BMN 110 is administered to MPS IVA patients by IV infusion, allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes. This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate (KS) in tissue macrophages, hyaline cartilage, other connective tissues, and heart valve, and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders.

Conditions

• MPS IV A

Interventions

Type	Name	Description
DRUG	BMN 110 Weekly	BMN 110 Weekly: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
DRUG	Placebo	Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week.
DRUG	BMN 110 Every Other Week	BMN 110 Every Other Week: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks.

Timeline

• Start date: 2011-02-01
• Primary completion: 2012-08-01
• Completion: 2012-08-01
• First posted: 2011-01-12
• Last updated: 2014-07-07
• Results posted: 2014-04-15

Locations

28 sites across 17 countries: United States, Argentina, Brazil, Canada, Colombia, Denmark, France, Germany, Italy, Japan, Netherlands, Portugal, Qatar, Saudi Arabia, South Korea, Taiwan, United Kingdom